Detalhe da pesquisa
1.
Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access.
Lancet
; 399(10322): 372-383, 2022 01 22.
Artigo
Inglês
| MEDLINE | ID: mdl-35065785
2.
Metachromatic leukodystrophy: A single-center longitudinal study of 45 patients.
J Inherit Metab Dis
; 44(5): 1151-1164, 2021 09.
Artigo
Inglês
| MEDLINE | ID: mdl-33855715
3.
Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial.
Lancet
; 388(10043): 476-87, 2016 Jul 30.
Artigo
Inglês
| MEDLINE | ID: mdl-27289174
4.
Alterations in the brain adenosine metabolism cause behavioral and neurological impairment in ADA-deficient mice and patients.
Sci Rep
; 7: 40136, 2017 01 11.
Artigo
Inglês
| MEDLINE | ID: mdl-28074903
5.
Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy.
Science
; 341(6148): 1233158, 2013 Aug 23.
Artigo
Inglês
| MEDLINE | ID: mdl-23845948